One of the world's top ex­perts in coro­nary heart dis­ease is spear­head­ing a new gene edit­ing up­start out to trans­form the field

As head of the Cen­ter for Hu­man Ge­net­ic Re­search at Mass­a­chu­setts Gen­er­al Hos­pi­tal and the Broad’s Car­dio­vas­cu­lar Dis­ease Ini­tia­tive, Sekar Kathire­san has oc­cu­pied a sin­gu­lar po­si­tion as one of the world’s lead­ing ex­perts on the con­nec­tion be­tween ge­net­ics and coro­nary heart dis­ease. He’s tracked how peo­ple dealt a bad ge­net­ic hand — and the el­e­vat­ed risks that come with it — can lim­it in­her­ent dan­gers by lifestyle changes, and pon­dered over the ef­fects of dai­ly drugs used to treat mass pa­tient groups. And he’s reached a sim­ple con­clu­sion:

None of it is re­al­ly work­ing. 

In par­tic­u­lar, none of that is any­where near as use­ful as the ge­net­ic mu­ta­tions that he’s seen that con­fer a low­er risk of dy­ing and car­dio events. Par­tic­u­lar­ly the in­di­vid­u­als who car­ry mu­ta­tions “which break ei­ther of two genes — APOC3 or ANGPTL3 — rapid­ly clear triglyc­eride-rich lipopro­teins from the cir­cu­la­tion” and pro­tect them from heart at­tack.

Now, he’s mak­ing a pro­fes­sion­al leap to see if he and a squad of in­ves­ti­ga­tors at a new­ly launched biotech can dra­mat­i­cal­ly change the im­per­fect sta­tus quo through gene edit­ing.

“Imag­ine,” he tells me, “an in­jec­tion ad­min­is­tered once in life that safe­ly con­fers last­ing pro­tec­tion.”

No more pills. No fleet­ing com­mit­ments to healthy lifestyles that can’t stretch past Jan­u­ary. But a wide­spread and durable shar­ing of the same health ben­e­fits he’s seen in the very, very few. That’s the dream.

To­day, Kathire­san is step­ping down from his lofty aca­d­e­m­ic roles and mak­ing his de­but as CEO of Verve Ther­a­peu­tics, which is tak­ing its place in the hotbed of gene ther­a­py work around Cam­bridge, MA. The small team of 10 — soon to dou­ble in size — may not come close to ri­val­ing the big biotechs that oc­cu­py the streets in and around Har­vard and MIT. But rel­a­tive­ly few can claim the same kinds of con­nec­tions in the realms of drug sci­ence.

Ki­ran Musunuru

An­oth­er car­dio ge­net­ics ex­pert, Penn’s Ki­ran Musunuru and Har­vard pro­fes­sor J Kei­th Joung, who co-found­ed gene ther­a­py pi­o­neer Ed­i­tas, are on board as sci­en­tif­ic co-founders. There’s an al­liance with Beam Ther­a­peu­tics, the up­start next-gen gene edit­ing out­fit found­ed by Feng Zhang, one of 3 sci­en­tists wide­ly cred­it­ed with ush­er­ing in the CRISPR rev­o­lu­tion that has armed re­searchers around the world with ef­fec­tive tools to ac­com­plish their work. And Ver­i­ly will help work on the nanopar­ti­cles they plan to use for de­liv­ery.

Burt Adel­man, the for­mer EVP of R&D at Bio­gen, will chair the board, which in­cludes the Broad’s chief da­ta of­fi­cer, An­tho­ny Philip­pakis.

Beam will pro­vide some of the tech, and has an op­tion to step in on fu­ture com­mer­cial­iza­tion. Verve has al­so nailed down CRISPR patents, in­clud­ing Cas9 and Cas12a (Cpf1), from the Broad In­sti­tute and Har­vard Uni­ver­si­ty.

And they have $58.5 mil­lion in cash to do their work from GV (you prob­a­bly still think of them as Google Ven­tures), which is step­ping in with ARCH Ven­ture Part­ners, F-Prime Cap­i­tal, and Bio­mat­ics Cap­i­tal to form the orig­i­nal syn­di­cate.

To be suc­cess­ful, the Verve team un­der Kathire­san will not just have to demon­strate their ap­proach can safe­ly work, but al­so that it ul­ti­mate­ly can be done on a mass ba­sis in eco­nom­ic terms. That’s a tall or­der, but they do have some ad­van­tages, per­haps most no­tice­ably the ad­vances the ground­break­ers have made at the FDA, says the sci­en­tist.

“Gene edit­ing has the po­ten­tial to com­plete­ly trans­form the treat­ment par­a­digm for the dis­ease,” says Musunuru. “Pre­clin­i­cal stud­ies con­duct­ed in the field, in­clud­ing work done in my lab, have shown the promise of gene edit­ing to safe­ly re­duce cho­les­terol and oth­er coro­nary artery dis­ease risk fac­tors.”

So far, gene edit­ing in the le­git­i­mate R&D world has been cen­tered on the painstak­ing ad­vances of a hand­ful of pro­grams aimed at rare dis­eases. And Verve will start with its own rare ail­ments, tar­get­ing pa­tient pop­u­la­tions with the high­est un­met med­ical need. But Kathire­san isn’t drop­ping his pres­ti­gious aca­d­e­m­ic roles to search for mar­gin­al gains. He wants to tack­le the whole threat on a world­wide ba­sis.

That, of­fi­cial­ly, starts at Verve to­day.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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J&J ad­comm live blog: Com­mit­tee votes 22-0 to rec­om­mend an FDA OK for the J&J vac­cine, set­ting up 3rd US Covid-19 jab

The US could have a third authorized Covid-19 vaccine within hours.

The FDA’s advisory committee voted unanimously — 22-0 — to recommend the agency issue an emergency use authorization for J&J’s vaccine. If they follow the precedent of the Pfizer and Moderna vaccine,  the FDA will likely authorize the vaccine by Saturday, immediately adding a few million doses to the US supply and adding a 100 million by June. An authorization would give the world its first single-dose vaccine, a major weapon in the effort to vaccinate the world and bring the virus to heel, particularly in rural and developing areas.